This phase II trial is studying how well fenretinide works in treating children with recurrent or resistant neuroblastoma. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.
OBJECTIVES: Determine the response rate in pediatric patients with recurrent or resistant high-risk neuroblastoma treated with fenretinide. Determine the toxic effects of this drug in these patients. Determine the proportion of patients with disease detected only by bone marrow immunocytology, who clear all evidence of disease during treatment with this drug. Determine minimal residual disease response by marrow and meta-iodobenzylguanidine (MIBG) I 123 scan in patients treated with this drug. OUTLINE: Patients are stratified according to presence of measurable disease on CT scan/MRI (yes vs no). A third stratum of patients with tumor cells in bone marrow by immunocytology only is enrolled but is not evaluated for response. Patients receive oral fenretinide 3 times daily (or 2 times daily if over 18 years of age) on days 1-7. Treatment repeats every 3 weeks for up to 30 courses in the absence of disease progression or unacceptable toxicity. Patients in stratum III who fail to achieve a complete response after 8 courses of therapy are removed from study. Patients are followed monthly until blood counts and visual acuity are stable or normalized and then every 6 months for 2 years and annually for 3 years. PROJECTED ACCRUAL: A total of 70 patients (25 each for strata I and II, 20 for stratum III) will be accrued for this study within 1-2 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
70
Given orally
Optional correlative studies
Children's Oncology Group
Arcadia, California, United States
Response rate
A responder is defined to be a patient who achieves a best overall response of complete response (CR), very good partial response (VGPR) or partial response (PR).
Time frame: Up to 8 courses of therapy
Toxicity as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 3.0
Assessed via a descriptive tabulation of the toxicity rates, overall and by stratum.
Time frame: Up to 5 years
Levels of fenretinide
Assessed via descriptive analysis of the steady state levels of fenretinide overall and by stratum
Time frame: At baseline and during courses 1, 2, and 5
Plasma retinol levels
Assessed via descriptive analysis of the plasma retinol levels overall and by stratum.
Time frame: At baseline and during courses 1, 2, and 5
Minimal residual disease (MRD) (Stratum 3)
Assessed by descriptive calculation of the proportion of responders.
Time frame: Up to 5 years
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